Overview

Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy

Status:
Completed
Trial end date:
2010-02-01
Target enrollment:
0
Participant gender:
All
Summary
This study is meant to assess the effectiveness of idebenone on visual function measures in patients with Leber's Hereditary Optic Neuropathy over a 6 months period.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Santhera Pharmaceuticals
Treatments:
Idebenone
Ubiquinone
Criteria
Inclusion Criteria:

- Age > or = 14 years and < 65 years

- Impaired visual acuity in at least one eye due to LHON

- Onset of visual loss due to LHON lies five years or less prior to Baseline

- Confirmation of either G11778A, T14484C or G3460A LHON mtDNA mutations at >60% in
blood

- No explanation for the visual failure besides LHON

- Body weight ≥ 45 kg

- Negative urine pregnancy test at Screening and at Baseline (women of childbearing
potential).

Exclusion Criteria:

- Treatment with Coenzyme Q10 or idebenone within 1 month prior to Baseline

- Pregnancy and/or breast-feeding

- Weekly alcohol intake 35 units (men) or 24 units (women)

- Current drug abuse

- Clinically significant abnormalities of clinical haematology or biochemistry
including, but not limited to, elevations greater than 2 times the upper limit of
normal of AST, ALT or creatinine

- Participation in another clinical trial of any investigational drug within 3 months
prior to Baseline

- Other factor that, in the investigator's opinion, excludes the patient from entering
the study